Boston– The U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation to enGene’s lead investigational treatment, detalimogene voraplasmid, for patients with high-risk, non-muscle invasive bladder cancer (NMIBC) that no longer responds to Bacillus Calmette-Guérin (BCG) therapy.
The designation, intended for promising regenerative treatments addressing serious or life-threatening conditions, could accelerate detalimogene’s path to approval. It provides enGene with expanded access to FDA support, including more frequent interactions, rolling submission of data, and the potential for priority review.
Detalimogene, formerly known as EG-70, is being evaluated in the ongoing LEGEND trial. Preliminary results from the study suggest the therapy is both clinically active and generally well tolerated in patients with carcinoma in situ (CIS) who have few alternatives beyond invasive surgery.
Unlike traditional gene therapies, detalimogene uses a non-viral platform to stimulate a local immune response in the bladder. enGene says the treatment can be administered in standard urology settings — including community clinics, where the majority of bladder cancer care occurs — potentially improving accessibility for patients.
“This designation reflects the encouraging early data and the urgent need for new bladder cancer treatments,” said enGene CEO Ron Cooper. “Patients facing limited options can’t afford to wait, and this brings us a step closer to offering a less invasive alternative to surgery.”
Bladder cancer remains one of the most challenging urologic cancers to treat when it becomes unresponsive to BCG. The RMAT designation signals that the FDA views detalimogene as a candidate with significant therapeutic promise.
